Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Immune Thrombocytopenia (ITP)

NCT01443351 | Withdrawn

• 1 other identifier: TPO-SJ-227
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Thrombopoietin Receptor Agonists (TPO-ra) are novel treatment modalities for patients with refractory Primary Immune Thrombocytopenia (ITP), but only few data are available for long-term effects of these drugs. In this observational study, effects and adverse effects including evaluation of bone marrow biopsies done at fixed intervals will be recorded from ITP patients treated with TPO-ra. For some patients, blood samples will be collected for research use.

Conditions

Idiopathic Thrombocytopenic Purpura

Keywords

ITP; romiplostim; eltrombopag; bone marrow fibrosis; long-term safety

Outcome Measures

Primary Outcomes (1)

• Number of patients with sustained response at 6 months follow-up

  Response is defined as platelet count \> 30.000 mio/L and no bleeding

  6 months

Secondary Outcomes (4)

• Yearly response rates

  5 years

• Frequency of relapse

  5 years

• Rate of splenectomy

  5 years

• Development of reticulin fibrosis in bone marrow biopsies

  5 years

Study Arms (1)

ITP patients

Patients with refractory ITP eligible for treatment with TPO-ra

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patinets will be selected from Departments of Hematology

You may qualify if:

• Adult patients with ITP
• platelet counts \< 25 x10\*9/L or \< 50 x10\*9/L with bleeding symptoms
• meets criteria for treatment with TPO-ra

You may not qualify if:

• Pregnancy or nursing
• Former thromboembolic events excluding one incidence of deep venous thrombosis as complication to surgery or pregnancy or one incidence of cerebral embolism as complication to atrial fibrillation
• Liver insufficiency (for eltrombopag only)
• TPO-ra contraindications (e.g. allergy)

Sponsors & Collaborators

• Zealand University Hospital: lead
• Rigshospitalet, Denmark: collaborator
• Odense University Hospital: collaborator

Study Sites (1)

Copenhagen University Hospital Roskilde

Roskilde, 4000, Denmark

Location

Biospecimen

Retention: SAMPLES WITH DNA

Whole blood samples will be collected, seru, plasma and mononuclear cells will be stored in a biobank for further research use

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Primary Myelofibrosis

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Myeloproliferative Disorders; Bone Marrow Diseases

Study Officials

• Hans Hasselbalch, Prof., MD

  Department of Hematology, Copenhagen University Hospital Roskilde

  PRINCIPAL INVESTIGATOR

• Henrik Frederiksen, Dr., MD

  Odense University Hospital

  PRINCIPAL INVESTIGATOR

• Sif Gudbrandsdottir, MD

  Copenhagen University Hospital Roskilde

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: September 13, 2011
• First Posted: September 29, 2011
• Study Start: March 1, 2015
• Primary Completion: August 1, 2019
• Study Completion: August 1, 2019
• Last Updated: September 11, 2019

Record last verified: 2019-09

Locations

DK (1)